(AIM: AMYT ) said the European Medicines Agency ("EMA") Committee for Orphan Medicinal Products ("COMP") has adopted a positive opinion for orphan designation for the use of its preclinical development, AP103, in Epidermolysis Bullosa ("EB").
The commercial-stage biopharmaceutical group focused on serious and life-threatening rare diseases said AP103 is based on its gene-therapy platform technology and offers a potential treatment for patients with Dystrophic Epidermolysis Bullosa ("DEB"), a subset of EB.
DEB is an incurable, inherited, and often fatal skin blistering skin disorder caused by a lack of collagen protein in the skin. This makes the skin incredibly fragile, leading to blistering or skin loss at the slightest friction or knock. It is both progressive and incredibly painful.
Orphan drug designation in the EU is granted by the European Commission within 30 days of a positive opinion being issued by the EMA COMP. The designation gives regulatory and financial incentives such as product market exclusivity for 10 years in the EU once approved.
Orphan designation is available for those developing treatments for a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the EU.
Amryt told investors that it plans to submit an orphan drug designation request for AP103 to the US Food and Drug Administration ("FDA") ‘in the near future.’
Shares in Amryt Pharma have climbed from lows of 155p in the past week and were trading 8.50% higher at 217p this morning following the announcement.
"Receiving this positive opinion from the EMA for our gene-therapy candidate, AP103, is a significant development for patients suffering from Epidermolysis Bullosa and provides additional momentum to our development pipeline,” said CEO, Dr Joe Wiley.
AP103 is the first gene therapy product candidate based on our novel polymer-based topical gene-therapy delivery platform, which also has potential use for the treatment of other rare genetic diseases. Amryt intends to initiate AP103’s clinical development in 2022’s first half.
He added, “Unlike other gene therapies that rely on viral vectors, AP103 is based on a novel polymer-based topical delivery platform, which we believe offers potential advantages in the gene-therapy field and has potential to be used in other genetic skin conditions.”
“Alongside our recent positive top line readout from our Phase 3 EASE trial in FILSUVEZ®, today's news further illustrates the exciting clinical opportunities in Amryt's portfolio.
These milestones relating to our efforts in Epidermolysis Bullosa are significant for all Amryt stakeholders as we build a global Epidermolysis Bullosa franchise and become a leader in rare and orphan diseases,” he said.
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